I cover cardiology news for CardioExchange, a social media website for cardiologists published by the New England Journal of Medicine. I was the editor of TheHeart.Org from its inception in 1999 until December 2008. Following the purchase of TheHeart.Org by WebMD in 2005, I became the editorial director of WebMD professional news, encompassing TheHeart.Org and Medscape Medical News. Prior to joining TheHeart.Org, I was a freelance medical journalist and wrote for a wide variety of medical and computer publications. In 1994-1995 I was a Knight Science Journalism Fellow at MIT. I have a PhD in English from SUNY Buffalo, and I drove a taxicab in New York City before embarking on a career in medical journalism. You can follow me on Twitter at: @cardiobrief.

FDA Approves Mipomersen For Homozygous Familial Hypercholesterolemia

The FDA said today that it had granted approval to the novel cholesterol-lowering drug mipomersen sodium for use as an adjunct to diet and drug therapy in patients with homozygous hypercholesterolemia. The drug, which was developed by Isis Pharmaceuticals, will be marketed under the brand name of Kynamro by Genzyme.

Kynamro was approved as an orphan drug, which the FDA describes as meaning it was developed to treat a condition affecting less than 200,000 people. The standard of approval for orphan drugs is less rigorous than other drugs. There has been some confusion about the number of people who have homozygous familial hypercholesterolemia. The FDA and ISIS/Genzyme said the condition affects about one in every million people.

In December the FDA approved lomitapide (Juxtapid, Aegerion) for the same condition. These are the first drugs specifically developed and approved for familial hypercholesterolemia.

Kynamro will carry a boxed warning about the serious risk of liver toxicity. Clinical studies with the drug found that it raised liver enzymes and led to accumulation of fat in the liver. The FDA said this “could lead to progressive liver disease with chronic use.”

Kynamro was approved with a Risk Evaluation and Mitigation Strategy (REMS) which will require certification of prescribers and pharmacies, as well as documentation that the drug is being properly used with each new prescription.

The FDA said the most common adverse reactions with Kynamro were injection site reactions, flu-like symptoms, nausea, headache and elevations in liver enzymes.

The FDA is requiring the sponsor to perform four postmarketing studies, including a long-term registry of patients to assess the long-term safety of the drug, and an enhanced pharmacovigilance program to monitor potential problems.

“Kynamro is the first systemic antisense drug to reach the market and is the culmination of two decades of work to create a new, more efficient drug technology platform. As evidenced by our robust pipeline, our antisense drug discovery technology is applicable to many different diseases, including the treatment of a chronic and rare disease, like HoFH,” said Stanley Crooke, the chair and CEO of Isis, in a press release.

The U.S. Food and Drug Administration today approved Kynamro (mipomersen sodium) injection as an addition to lipid-lowering medications and diet to treat patients with a rare type of high cholesterol called homozygous familial hypercholesterolemia (HoFH). The addition of Kynamro helps to reduce low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B, total cholesterol, and non-high density lipoprotein-cholesterol (non HDL-C).

HoFH, an inherited condition that affects about one out of every one million people in the United States, occurs when the body is unable to remove LDL-C, often called “bad” cholesterol, from the blood causing abnormally high levels of circulating LDL-C. For those with HoFH, heart attacks and death often occur before age 30. Kynamro is an orphan drug approval, meaning it was developed to treat a disorder affecting fewer than 200,000 people. In December 2012, the FDA approved Juxtapid (lomitapide) to reduce LDL-C, total cholesterol, apolipoprotein B, and non HDL-C in patients with HoFH.

“Kynamro, an injection given once a week, works with other lipid-lowering medications and diet to impair the creation of the lipid particles that ultimately give rise to LDL-C,” said Eric Colman, M.D., deputy director of the Division of Metabolism and Endocrinology Products at the FDA’s Center for Drug Evaluation and Research.

The safety and effectiveness of Kynamro were evaluated in a clinical trial of 51 patients with HoFH. On average, levels of LDL-C fell by about 25 percent during the first 26 weeks in those receiving the drug. Kynamro carries a Boxed Warning on the serious risk of liver toxicity because it is associated with liver enzyme abnormalities and accumulation of fat in the liver, which could lead to progressive liver disease with chronic use.

The FDA approved Kynamro with a Risk Evaluation and Mitigation Strategy (REMS) with elements to assure safe use, including prescriber and pharmacy certification, and documentation of safe-use conditions, which requires a prescription authorization form for each new prescription.

The most common adverse reactions in the clinical trial included injection site reactions, flu-like symptoms, nausea, headache and elevations in liver enzymes (serum transaminases).

The FDA is requiring four postmarketing studies for Kynamro: the development of a sensitive assay that binds double-stranded (ds) DNA; a study to assess for the presence of antibodies to ds-DNA in patients treated with Kynamro; a long-term registry of patients with HoFH to determine the long-term safety of Kynamro; and an enhanced pharmacovigilance program to monitor reports of malignancy, immune-mediated reactions, and hepatic abnormalities in patients treated with Kynamro.

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